M. Scott Perry, MD

Overview of Dravet Syndrome

Importance of Early Diagnosis of Dravet Syndrome  

Treatment Options for Dravet Syndrome

Approval of Cannabidiol and Fenfluramine for Dravet

Choosing Cannabidiol or Fenfluramine for Dravet

Future Outlook for Dravet Syndrome

Videos

Scott Perry, MD, reviews the latest advances in the management of Dravet syndrome. 

Advances in the Management of Dravet Syndrome

“This is a superior drug for Dravet syndrome. The seizure reductions I’m seeing, especially with convulsive seizures, in these kids is as significant as the phase 3 data.”

American Academy of Neurology (AAN) 2020 Annual Meeting

 data remains abound in the neurology space. One such dataset which was scheduled to be presented at AAN 2020 was that of the first 23 patients in the fenfluramine (Fintepla; Zogenix) Expanded Access Program (EAP), which was collected by M. Scott Perry, MD, medical director of neurology, and co-director, Jane and John Justin Neurosciences Center, Cook Children’s Hospital, and colleagues.

, and these real-world data appear to confirm the reductions in seizure frequency that were observed in the pivotal clinical trials. Now, however, with further context of its use, 

 reached out to Perry to find out more about his experience with fenfluramine ahead of possible agency approval.

Perry highlighted some of the success that patients of his have had with the agent being used in combination with other therapies such as clobazam, valproate, stiripentol, and cannabidiol (CBD) through the EAP, as well as what other aspects of its use he would like to see explored in future assessments of the drug.

Perry MS, Wirrell E, Burkholder D, Galer BS, Gammaitoni A. Real-world Experience with ZX008 (Fenfluramine HCl) for the Treatment of Seizures in Dravet Syndrome: Initial Report from United States Expanded Access Program. 

The co-director of the Jane and John Justin Neurosciences Center at Cook Children’s Hospital discussed his experience with Zogenix’s investigational Dravet syndrome treatment in clinical trials and in the Expanded Access Program.

M. Scott Perry, MD: Clinical Experience With Fenfluramine in Dravet Syndrome

“When we start the medicine, we keep it stable for 30 days before we start making changes in other meds, but we can make changes in other meds and I think that’s really the take-home [message].”

New real-world data on fenfluramine (Fintepla; Zogenix) that were accepted for presentation at the 

 suggest that similarly to the clinical trials, the therapy results in clinically meaningful responses in patients with Dravet syndrome.

Conducted by M. Scott Perry, MD, medical director of neurology, and co-director, Jane and John Justin Neurosciences Center, Cook Children’s Hospital, and colleagues, this initial report of the US Expanded Access Program for fenfluramine included 23 patients with Dravet syndrome with a mean age of 6.8 years, who had been treated for a median of 90 days. In total, 65% (n = 15) of the cohort reported meaningful global clinical improvement, defined as a rating of “very much improved” or “much improved.” The most common concomitant antiepileptic drugs (AEDs) were clobazam, valproate, stiripentol, and cannabidiol (CBD).

, these data mean quite a bit for those who are treating this patient population. In order to find out more about these data which were sent to be presented at AAN and what the clinical community needs to know, 

 caught up with Perry virtually to discuss.

The co-director of the Jane and John Justin Neurosciences Center at Cook Children’s Hospital discussed the real-world data collected from the Expanded Access Program for fenfluramine (Fintepla; Zogenix) in patients with Dravet syndrome.

M. Scott Perry, MD

Articles

Advances in the Management of Dravet Syndrome

M. Scott Perry, MD: Clinical Experience With Fenfluramine in Dravet Syndrome

M. Scott Perry, MD: Real-World Fenfluramine Data in Dravet Syndrome